2025 Rally for Rare: Bringing the Rare Disease Community Together

In a recent video interview with Applied Clinical Trials, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, discussed the unique challenges in rare disease R&D, including competing priorities among researchers, difficulty in diagnosing and treating patients, and defining endpoints due to varying symptoms. Ansel also touched on the potential of technology and artificial intelligence to address some of these challenges and improve rare disease research.

ACT: Could you tell our audience a bit about the Rally for Rare event and its importance in raising awareness around rare disease research?

Ansel: Our Rally for Rare event was a last-minute event, I will say. We were very much looking forward to attending the combined NIH-FDA, Rare Disease Day event in Bethesda that was postponed, and that event is so important, one, because it is an avenue for both patients, caregivers, those affected in the rare disease community to collaborate directly with researchers and physicians at NIH, at FDA, and other various scientific consortium and it really sets the precedent for global engagement in rare disease research. It's quite a remarkable event, so the fact that the event was postponed, really represented a gap for that collaboration. The work that these institutions do for rare disease research is critical and having that platform to discuss all of the things that I've mentioned thus far, a commitment around global awareness and engagement, commitment on endpoints, and removing those barriers and removing the red tape to drive rare disease research is really the pinnacle of that event, so Rally for Rare was our response to continuing that conversation, allowing those who otherwise would have been at that event to come and share in that space the same platform discussion and innovation they would have had at that event. It was held on Rare Disease Day, February 28, and we welcomed everybody in the rare disease community, patients, caregivers, advocacy groups, researchers, those of us in the industry to again, to continue that conversation. We use the term “rally for rare” to again, represent just how important this day is, Rare Disease Day, but also just because the event was being postponed to a later date in the year, that it was still important to have that conversation on that day for all the various reasons that I've mentioned.